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Simply Stated: Updates in Neuromuscular Junction (NMJ) Disorders
The neuromuscular junction (NMJ) disorders are a group of conditions that disrupt the communication between motor […]
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Clinical Research Opportunity: LION-CS101 a Phase 1/2 Study of AB-1003 in Adults with LGMD2I/R9
Researchers at AskBio are seeking adults with genetically confirmed limb-girdle muscular dystrophy (LGMD) type 2I/R9 to […]
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Dr. Peter Marks Resigns from FDA Center for Biologics Evaluation and Research
PPMD is deeply disappointed that Dr. Peter Marks has made the decision to leave his […]
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Pro Tips to Add More Movement to Your Day
Adding more movement or physical activity to each day is a common goal among people […]
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MDA 2025: Duvyzat delays walking loss, lessens lung function decline
Long-term treatment with Duvyzat (givinostat) may help delay the loss of walking ability and lessen […]
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Wave Life Sciences Announces Positive Data from FORWARD-53 Clinical Trial
Wave Life Sciences has announced encouraging interim results from its Phase 2 FORWARD-53 clinical trial […]
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A virtual art show gave FSHDers a chance to show off our talents
I was tempted to do a play on the show “America’s Got Talent” and title […]
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Target to repair injured muscles discovered
A team has discovered a possible therapeutic approach to repair injured muscles either from aging […]
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MDA 2025: Donavon Decker honored for decades of advocacy
Patient advocate Donavon Decker has been given the 2025 MDA Legacy Award for Community Impact […]
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REGENXBIO Shares Positive Biomarker Data from AFFINITY DUCHENNE Trial of RGX-202 Gene Therapy
REGENXBIO Inc. has reported new, positive interim data from two additional patients in the Phase […]
